RARE DISEASE
Lead poisoning
Lead poisoning
Lead poisoning
Synonyms: Lead intoxication, Plumbism, Saturnism
Synonyms: Lead intoxication, Plumbism, Saturnism
Synonyms: Lead intoxication, Plumbism, Saturnism
Drug discovery
3
drugs
With orphan designations
Overview
Lead poisoning is a systemic toxicosis caused by lead accumulation, primarily affecting neurological development in children and cardiovascular/renal function in adults. Exposure occurs through lead-based paint, contaminated soil/water, and occupational sources. Diagnosis relies on venous blood lead levels (BLL), with treatment focused on exposure cessation and chelation therapy for BLL ≥45 μg/dL. Neurocognitive effects are often irreversible, emphasizing prevention [1][6][15][16].
Population
Children <6 years account for 80% of global cases due to hand-to-mouth behavior and higher absorption rates [2][9]
High-risk groups: Residents of pre-1978 housing, battery/recycling workers, and communities with poor lead regulation [7][12][17]
800 million+ children globally have BLL ≥5 μg/dL; 30x higher prevalence in low-income countries [9][14]
Burden
Therapies
Primary intervention: Source identification/removal + nutritional optimization (iron/calcium) [6][8][11]
Chelation 1) Oral DMSA for BLL 45-69 μg/dL 2) IV CaNa₂EDTA for BLL ≥70 μg/dL or encephalopathy [3][13][16]
Monitoring: Weekly BLL checks during chelation + environmental hazard mitigation [6][11]
Categories: rare disorders due to toxic effects
Research Papers
1,466 drug discovery papers about Lead poisoning, with 1 first-in-class and 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,466 drug discovery papers about Lead poisoning, with 1 first-in-class and 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-15 | Groundnut Shell-Derived Activated Carbon for Lead (Ii) Removal from Aqueous Solution: A Low-Cost Approach to Mitigating Lead Poisoning
Lead (Pb) contamination is a matter of great concern due to its toxic, non-biodegradable and accumulative nature. In this study, activated carbon was prepared from groundnut shell waste by carbonization at 450 °C followed by chemical activation using phosphoric acid at 500 °C and evaluated for its Pb(II) removal capacity from an aqueous solution. The removal mechanism and effectiveness of the adsorbent as a low-cost adsorbent were determined using batch adsorption experiments. The effects of adsorbent doses (0.10-1.00 g/100mL) and solution pH (2.0-8.0) on the removal of Pb(II) were investigated. It was observed that the removal of Pb(II) increased with an increase in the adsorbent dose and attained maximum removal at 1.00g/100mL with a value of 96.4%. The removal of Pb(II) by the adsorbent was strongly dependent on the initial solution pH as the removal increased with an increase in solution pH from 2 to 6, after which it decreased moderately at pH 8 due to the hydrolysis of Pb(II). The removal capacity of GSAC in this study was comparable and in some cases higher than those reported in the literature for other agricultural wastes such as rice husk and coconut shell activated carbon. Overall, the results obtained show the potential of using groundnut shell waste as a low-cost and easily available adsorbent for the removal of Pb(II). Thus concluding that the utilization of the agricultural waste mentioned above would provide an alternative to the high-cost adsorbents currently used and help in reducing the lead content in water, hence minimizing its harmful effects on health.
2026-07-20 | Caffeic Acid Mitigates Behavioral and Biochemical Alterations in Lead-induced Neurotoxicity in Rats With Possible Involvement of TFEB.
Lead (Pb) neurotoxicity is characterized by persistent cognitive and motor impairments that arise from converging disturbances in mitochondrial function, oxidative balance, and neuroinflammatory signaling. Increasing evidence suggests that these pathological outcomes are closely linked to disruption of Transcription Factor EB (TFEB), a key regulator of autophagy-lysosomal pathways and mitochondrial quality control. Impaired TFEB function can promote the accumulation of dysfunctional mitochondria and perpetuate oxidative and inflammatory cascades, thereby exacerbating Pb-induced neurodegeneration. The present study evaluated whether caffeic acid (CFA) protects against Pb-induced neurotoxicity and investigated the involvement of TFEB signaling in its neuroprotective effects. Wistar rats were exposed to lead acetate (100 mg/kg, p.o.) for 30 days, followed by treatment with caffeic acid (CFA; 20 and 40 mg/kg). To validate the mechanistic role of TFEB, eltrombopag, a TFEB inhibitor, was co-administered in dedicated groups. Behavioral outcomes were assessed using the Morris Water Maze and rota rod tests, and hippocampal and cerebellar tissues were examined for mitochondrial complex I-III activities, oxidative stress indices (TBARS, GSH), and inflammatory mediators (TNF-α, IL-1β, NF-κB). Pb exposure produced marked spatial memory deficits, motor impairment, suppression of mitochondrial complex activities, oxidative imbalance, and enhanced inflammatory signaling. CFA treatment attenuated these alterations; however, these benefits were lost upon TFEB inhibition. Collectively, these findings demonstrate that CFA attenuates Pb-induced behavioral and biochemical alterations and suggest that its neuroprotective effects are mediated, at least in part, through TFEB-associated pathways.
2026-07-15 | THE EFFECT OF CHRONIC LEAD INTOXICATION ON BONE TISSUE METABOLISM AND OSTEOREPARATION: EXPERIMENTAL RATIONALE FOR COMBINED CORRECTIVE THERAPY
<p>Background. Lead is one of the most osteotropic toxicants: up to 90% of the total body burden is deposited within the bone mineral matrix, where it competitively substitutes for Ca2+ in the hydroxyapatite lattice, suppresses osteoblast differentiation and enhances osteoclastogenesis. A pathogenetically grounded correction scheme for lead-induced bone metabolic disturbances, and its relationship to reparative osteogenesis, remain insufficiently studied. Aim — to experimentally assess biochemical bone metabolism markers and reparative osteogenesis under chronic lead intoxication, and to evaluate the efficacy of combined corrective therapy (vitamin D3 + calcium citrate + succinic acid). Materials and methods. Forty-five rats were allocated to three groups (control, Pb-intoxication, Pb-intoxication + correction). Chronic intoxication was induced by lead acetate administered ad libitum via drinking water for 8 weeks; at week 4 a monocortical femoral defect was created in all animals. Serum biochemical markers and cortical bone lead content (ICP-OES) were assessed. Results. The Pb group showed significantly reduced osteoblastic activity (alkaline phosphatase −39%, osteocalcin −54%), hypocalcemia (−25%) and marked bone lead accumulation (244.1±21.0 µg/g). Corrective therapy reduced bone lead deposition by 62% and produced statistically significant partial recovery of the studied biochemical parameters. Conclusion. Chronic lead intoxication produces a complex osteotoxic syndrome with disturbed biochemical markers of reparative osteogenesis; the proposed corrective regimen is pathogenetically justified and may serve as a basis for further clinical development of osteoprotective protocols.</p>
2026-07-12 | Lead Poisoning: Bridging Historical Perspectives, Contemporary Toxicology, and Management Strategies
Lead poisoning represents one of the oldest and most persistent environmental health challenges confronting human civilization, spanning millennia of recognized toxicity yet remaining incompletely resolved in contemporary practice. This comprehensive review examines the multifaceted dimensions of plumbism, from its ancient recognition as a neurotoxic hazard to the modern understanding of its subclinical effects at vanishingly low blood lead concentrations. The etiology of lead exposure encompasses legacy sources such as deteriorating lead-based paint and contaminated plumbing, occupational and avocational hazards, and less ubiquitous but clinically significant exposures from imported goods and traditional remedies. Epidemiological surveillance reveals that hundreds of thousands of children in the United States continue to harbor elevated blood lead concentrations, with disproportionate burden falling upon socioeconomically disadvantaged communities residing in older housing stock. The pathophysiology of lead toxicity is extraordinarily complex, involving interference with sulfhydryl-containing enzymes, disruption of divalent cation homeostasis, and multifaceted organ system dysfunction affecting the neurologic, hematologic, renal, cardiovascular, and endocrine systems. The toxicokinetic profile of lead is characterized by differential absorption, distribution, and elimination in children compared to adults, fundamentally explaining the heightened pediatric vulnerability to this cumulative toxin. Clinical manifestations span a spectrum from asymptomatic elevation to the catastrophic syndrome of acute lead encephalopathy, with the most concerning public health impact being the inverse relationship between blood lead concentration and cognitive function, manifested as measurable decrements in intelligence quotient at levels once considered innocuous. Management strategies emphasize the primacy of source identification and environmental remediation, with chelation therapy reserved for cases of severe toxicity under the guidance of medical toxicologists. The prognosis remains guarded, as many lead-induced neurologic sequelae appear irreversible despite intervention, underscoring the critical importance of primary prevention. Optimizing outcomes requires a coordinated interprofessional approach encompassing healthcare providers, public health officials, environmental specialists, educators, and social services, reflecting the complex interplay of biomedical, environmental, and social determinants that characterize this enduring public health challenge.
2026-07-05 | Ameliorative Effects of Ginkgo biloba on Lead Acetate-Induced Oxidative Stress and Histopathological Damages in Rat Liver, Kidney, and Testis.
Ginkgo biloba is a nutritional supplement known for its antioxidant properties and beneficial effects on different organs. This study aimed to investigate the protective effect of Ginkgo biloba supplementation against acute lead toxicity in rats. Thirty-two male Wistar rats were divided into four groups and treated daily for 7 days. The control group received normal saline (1 mL/day, orally). The model group received lead acetate (50 mg/kg/day, ip) without any treatment, and the model with either ethylenediaminetetraacetic acid (EDTA) (50 mg/kg/day, ip) or Ginkgo biloba (200 mg/kg/day, oral gavage). After 7 days, blood samples were collected, and livers, kidneys, and testes were excised and stored for evaluation of oxidative stress markers and histological assessment. Acute lead acetate administration resulted in significant weight loss, an increase in kidney index, elevated serum creatinine and liver enzyme levels, including serum glutamic oxaloacetic transaminase (SGOT), serum glutamic-pyruvic transaminase (SGPT), and alkaline phosphatase (AlkP), and extensive histological damage in the liver, kidney, and testicular tissues. Administration of the Ginkgo biloba significantly improved serum creatinine and AlkP, showing greater efficacy than EDTA, and reduced histopathological changes, similar to EDTA, including hepatic vascular congestion and fibrosis, renal dilation of Bowman spaces, and glomerular degeneration. Furthermore, Ginkgo biloba treatment improved Leydig and spermatogonia cell counts. Both Ginkgo biloba and EDTA reduced malondialdehyde (MDA) levels and enhanced superoxide dismutase (SOD) and catalase activities in these organs. Ginkgo biloba exhibited protective effects against lead-induced hepatic, renal and testicular damage through its ability to reduce oxidative stress damage. These results indicate that Ginkgo biloba may serve as a valuable adjunct therapy for mitigating lead-associated organ damage.
2026-08-15 | Groundnut Shell-Derived Activated Carbon for Lead (Ii) Removal from Aqueous Solution: A Low-Cost Approach to Mitigating Lead Poisoning
Lead (Pb) contamination is a matter of great concern due to its toxic, non-biodegradable and accumulative nature. In this study, activated carbon was prepared from groundnut shell waste by carbonization at 450 °C followed by chemical activation using phosphoric acid at 500 °C and evaluated for its Pb(II) removal capacity from an aqueous solution. The removal mechanism and effectiveness of the adsorbent as a low-cost adsorbent were determined using batch adsorption experiments. The effects of adsorbent doses (0.10-1.00 g/100mL) and solution pH (2.0-8.0) on the removal of Pb(II) were investigated. It was observed that the removal of Pb(II) increased with an increase in the adsorbent dose and attained maximum removal at 1.00g/100mL with a value of 96.4%. The removal of Pb(II) by the adsorbent was strongly dependent on the initial solution pH as the removal increased with an increase in solution pH from 2 to 6, after which it decreased moderately at pH 8 due to the hydrolysis of Pb(II). The removal capacity of GSAC in this study was comparable and in some cases higher than those reported in the literature for other agricultural wastes such as rice husk and coconut shell activated carbon. Overall, the results obtained show the potential of using groundnut shell waste as a low-cost and easily available adsorbent for the removal of Pb(II). Thus concluding that the utilization of the agricultural waste mentioned above would provide an alternative to the high-cost adsorbents currently used and help in reducing the lead content in water, hence minimizing its harmful effects on health.
2026-07-20 | Caffeic Acid Mitigates Behavioral and Biochemical Alterations in Lead-induced Neurotoxicity in Rats With Possible Involvement of TFEB.
Lead (Pb) neurotoxicity is characterized by persistent cognitive and motor impairments that arise from converging disturbances in mitochondrial function, oxidative balance, and neuroinflammatory signaling. Increasing evidence suggests that these pathological outcomes are closely linked to disruption of Transcription Factor EB (TFEB), a key regulator of autophagy-lysosomal pathways and mitochondrial quality control. Impaired TFEB function can promote the accumulation of dysfunctional mitochondria and perpetuate oxidative and inflammatory cascades, thereby exacerbating Pb-induced neurodegeneration. The present study evaluated whether caffeic acid (CFA) protects against Pb-induced neurotoxicity and investigated the involvement of TFEB signaling in its neuroprotective effects. Wistar rats were exposed to lead acetate (100 mg/kg, p.o.) for 30 days, followed by treatment with caffeic acid (CFA; 20 and 40 mg/kg). To validate the mechanistic role of TFEB, eltrombopag, a TFEB inhibitor, was co-administered in dedicated groups. Behavioral outcomes were assessed using the Morris Water Maze and rota rod tests, and hippocampal and cerebellar tissues were examined for mitochondrial complex I-III activities, oxidative stress indices (TBARS, GSH), and inflammatory mediators (TNF-α, IL-1β, NF-κB). Pb exposure produced marked spatial memory deficits, motor impairment, suppression of mitochondrial complex activities, oxidative imbalance, and enhanced inflammatory signaling. CFA treatment attenuated these alterations; however, these benefits were lost upon TFEB inhibition. Collectively, these findings demonstrate that CFA attenuates Pb-induced behavioral and biochemical alterations and suggest that its neuroprotective effects are mediated, at least in part, through TFEB-associated pathways.
2026-07-15 | THE EFFECT OF CHRONIC LEAD INTOXICATION ON BONE TISSUE METABOLISM AND OSTEOREPARATION: EXPERIMENTAL RATIONALE FOR COMBINED CORRECTIVE THERAPY
<p>Background. Lead is one of the most osteotropic toxicants: up to 90% of the total body burden is deposited within the bone mineral matrix, where it competitively substitutes for Ca2+ in the hydroxyapatite lattice, suppresses osteoblast differentiation and enhances osteoclastogenesis. A pathogenetically grounded correction scheme for lead-induced bone metabolic disturbances, and its relationship to reparative osteogenesis, remain insufficiently studied. Aim — to experimentally assess biochemical bone metabolism markers and reparative osteogenesis under chronic lead intoxication, and to evaluate the efficacy of combined corrective therapy (vitamin D3 + calcium citrate + succinic acid). Materials and methods. Forty-five rats were allocated to three groups (control, Pb-intoxication, Pb-intoxication + correction). Chronic intoxication was induced by lead acetate administered ad libitum via drinking water for 8 weeks; at week 4 a monocortical femoral defect was created in all animals. Serum biochemical markers and cortical bone lead content (ICP-OES) were assessed. Results. The Pb group showed significantly reduced osteoblastic activity (alkaline phosphatase −39%, osteocalcin −54%), hypocalcemia (−25%) and marked bone lead accumulation (244.1±21.0 µg/g). Corrective therapy reduced bone lead deposition by 62% and produced statistically significant partial recovery of the studied biochemical parameters. Conclusion. Chronic lead intoxication produces a complex osteotoxic syndrome with disturbed biochemical markers of reparative osteogenesis; the proposed corrective regimen is pathogenetically justified and may serve as a basis for further clinical development of osteoprotective protocols.</p>
2026-07-12 | Lead Poisoning: Bridging Historical Perspectives, Contemporary Toxicology, and Management Strategies
Lead poisoning represents one of the oldest and most persistent environmental health challenges confronting human civilization, spanning millennia of recognized toxicity yet remaining incompletely resolved in contemporary practice. This comprehensive review examines the multifaceted dimensions of plumbism, from its ancient recognition as a neurotoxic hazard to the modern understanding of its subclinical effects at vanishingly low blood lead concentrations. The etiology of lead exposure encompasses legacy sources such as deteriorating lead-based paint and contaminated plumbing, occupational and avocational hazards, and less ubiquitous but clinically significant exposures from imported goods and traditional remedies. Epidemiological surveillance reveals that hundreds of thousands of children in the United States continue to harbor elevated blood lead concentrations, with disproportionate burden falling upon socioeconomically disadvantaged communities residing in older housing stock. The pathophysiology of lead toxicity is extraordinarily complex, involving interference with sulfhydryl-containing enzymes, disruption of divalent cation homeostasis, and multifaceted organ system dysfunction affecting the neurologic, hematologic, renal, cardiovascular, and endocrine systems. The toxicokinetic profile of lead is characterized by differential absorption, distribution, and elimination in children compared to adults, fundamentally explaining the heightened pediatric vulnerability to this cumulative toxin. Clinical manifestations span a spectrum from asymptomatic elevation to the catastrophic syndrome of acute lead encephalopathy, with the most concerning public health impact being the inverse relationship between blood lead concentration and cognitive function, manifested as measurable decrements in intelligence quotient at levels once considered innocuous. Management strategies emphasize the primacy of source identification and environmental remediation, with chelation therapy reserved for cases of severe toxicity under the guidance of medical toxicologists. The prognosis remains guarded, as many lead-induced neurologic sequelae appear irreversible despite intervention, underscoring the critical importance of primary prevention. Optimizing outcomes requires a coordinated interprofessional approach encompassing healthcare providers, public health officials, environmental specialists, educators, and social services, reflecting the complex interplay of biomedical, environmental, and social determinants that characterize this enduring public health challenge.
2026-07-05 | Ameliorative Effects of Ginkgo biloba on Lead Acetate-Induced Oxidative Stress and Histopathological Damages in Rat Liver, Kidney, and Testis.
Ginkgo biloba is a nutritional supplement known for its antioxidant properties and beneficial effects on different organs. This study aimed to investigate the protective effect of Ginkgo biloba supplementation against acute lead toxicity in rats. Thirty-two male Wistar rats were divided into four groups and treated daily for 7 days. The control group received normal saline (1 mL/day, orally). The model group received lead acetate (50 mg/kg/day, ip) without any treatment, and the model with either ethylenediaminetetraacetic acid (EDTA) (50 mg/kg/day, ip) or Ginkgo biloba (200 mg/kg/day, oral gavage). After 7 days, blood samples were collected, and livers, kidneys, and testes were excised and stored for evaluation of oxidative stress markers and histological assessment. Acute lead acetate administration resulted in significant weight loss, an increase in kidney index, elevated serum creatinine and liver enzyme levels, including serum glutamic oxaloacetic transaminase (SGOT), serum glutamic-pyruvic transaminase (SGPT), and alkaline phosphatase (AlkP), and extensive histological damage in the liver, kidney, and testicular tissues. Administration of the Ginkgo biloba significantly improved serum creatinine and AlkP, showing greater efficacy than EDTA, and reduced histopathological changes, similar to EDTA, including hepatic vascular congestion and fibrosis, renal dilation of Bowman spaces, and glomerular degeneration. Furthermore, Ginkgo biloba treatment improved Leydig and spermatogonia cell counts. Both Ginkgo biloba and EDTA reduced malondialdehyde (MDA) levels and enhanced superoxide dismutase (SOD) and catalase activities in these organs. Ginkgo biloba exhibited protective effects against lead-induced hepatic, renal and testicular damage through its ability to reduce oxidative stress damage. These results indicate that Ginkgo biloba may serve as a valuable adjunct therapy for mitigating lead-associated organ damage.
Access all drug discovery papers and probability of success in trials forecasts:
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Drug Discovery Landscape
3 orphan drug designations for Lead poisoning, including 1 approved therapy.
3 orphan drug designations for Lead poisoning, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Erdosteine | small molecules | EMA | 2012-12-06 | — | Rafifarm SRL |
ALPHA-1-ACID GLYCOPROTEIN | — | EMA | 2003-10-02 | — | Bio Products Laboratory Limited |
Succimer [Chemet capsules] | small molecules | FDA | 1984-05-09 | 1991-01-30 | Bock Pharmacal Company |
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